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VRTX: I wish the AMRN ADCOM had panel members with integrity
FDA Halftime Show: Should Kalydeco Be Approved
in F508del CF patients?
Conclusion: FDA analysis hard on Vertex – but didn’t hold up to scrutiny
Today, the FDA is hosting an Advisory Committee regarding Vertex's Orkambi filing for
cystic fibrosis (CF) patients homozygous for the F508del mutation (LINK). The FDA
reviewers suggested there is a lack of evidence that lumacaftor adds efficacy to
Kalydeco alone. This was based on a cross-trial stat comparison, which modified the
agreed endpoints, as well as the inclusion criteria for the comparison. However, the
FDA review team quickly conceded under scrutiny that its analysis was half-baked and
that it now thinks its prior dismissal of Kalydeco in this population was "hasty" and
that it had advised Vertex to not include Kalydeco alone in the ph3 design. The final
committee expert comment prior to breaking for lunch was that this FDA stance would
penalize not only the sponsor (Vertex), but in particularly patients who could benefit
from the medicine. Hence despite the debate, we still think the panel will vote for
approval of the combination.
sts: You are correct, but the Outcomes Trial will
expand the label to include the very same indications
for Statins.
Kiwi; In Biotechnology the oft cited mantra is
'Cash is King" . AMRN's expenses are not keeping
up with sales of Vascepa. Rx's barely up from 2014,
and SG&A increasing steadily. Unless I'm missing
something, Costs will continue to increase, attorneys
fees, court costs, continuing R-IT, debt service,
(which seems to have been forgotten on this site)
and of course the out of control Administrative expenses.
A rational Mgmt would step up and address these issues.
The possibility of delays in R-IT results, FDA dilly dallying over final approval assuming the results are stellar
collectively imperil shareholders by the very possibility
that the Co runs out of cash. Hypothetically if R-IT is halted for efficacy, I'd love to see a BP partnership for the purpose
of doing a three arm Clinical Trial with their PCSK9 + Vascepa, vs PCSK9 alone and in combination with Statins. I'd bet on Vascepa
plus PCSK9. IMHO this would accelerate the removal of Stains
HD: Take a look at their pitiful balance sheet.
A bright guy like you should figure that out.
Question HD, Why does this Mgmt Team get full
pay checks, bonuses for everything , and anything,
stock grants, option grants etc, while imperiling
shareholders, with Cash and Cash equivalents , unable
to support the Co going forward. They have destroyed
hundreds of millions of dollars of shareholder value.
Oh, are you looking for another dilution ?
Kiwi: It's amazing that AMRN's financial woes are not addressed
by this pathetic Mgmt Team and BOD. Most other companies in the
sector would freeze salaries, bonuses, stock and option grants,
and other innovative cost cutting measures. I am still of the opinion that Mgmt should create shareholder value, not destroy
it. But I've always maintained that these guys are The Gang that
couldn't shoot straight. That is with the exception of aiming
at their own foot.
correction: That should read PCSK9
zum: Great question ! I'm not so sure it's about significantly
lowering Trigs. It's about decreasing CVE's , and Purified EPA
at 4gms a day lowers the key Inflammatory Markers. That IMO
is what lowers CVE's. It just may be that when the KCSK9's start
replacing Statins, then the combo will be KCSK9 + Vascepa. That
will represent Son of R-IT. another outcomes trail. The good news
is that AMRN will be doing well financially on Vascepa and statin
sales. The future may be that Vascepa causes Statins to be replaced by a combo with KCSK9. To answer your question, IMO Anchor will probably never be approved.
medtodamoon: They once asked Bernard Baruch, Wall Street Icon,
what he thought about stocks: His answer was "They fluctuate"
Hate is a negative emotion. Your pissed because you lost money.
If you're going to be angry, be angry with yourself, as you pulled
the trigger to buy and then to sell. My Rx for you is a tincture
of time.
BB: They are the mainstay of food inspection, beef
and poultry , CMC and GMP inspections and they, despite
all the noise, are completely backed by both houses of Congress.
You and I and others are angry at them and only wish a pox
upon their houses. Dismantling will never happen, certainly
not because of the AMRN issue. You're believing the last
3-4 years of your prolific anti FDA postings. Chill my friend,
all the kings Horses and all the Kings men wont change FDA
in a material way.
mrmainstreet: My take is that a decision for FDA would
be a short 3 page letter underscoring FDA's points.
If it's in favor of AMRN I would imagine that the Judge
is going to take his time, elucidate all his points and
make sure he dots all the I's and crosses all the T's
BB: You are looking at the FDA as if AMRN was it's only
task. They are up to their armpits in crocodiles, and
are far from ever being dismantled. FYI, I believe the
longer it takes the Judge to make a decision, the greater
likelihood that it will be in AMRN's favor.
BB: I NEVER ALLUDED TO THAT. FDA ADCOM WAS THE WORST I'VE
EVER WITNESSED. TAHT SAID YOU KEEP MISSING THE POINT.
CHILL PAL !
Zum: You verify my point about the ACA/AHA Guidelines not
believing there was proof that lowering Trigs would lower
CVE. Giving JL his due, lowering inflammatory markers is
the key to lowering CVE's . If we have a significant reduction,
25% or more, I wonder how the FDA will view the fact that
Trigs are not all that important in the scheme of things.
I've maintained that the change in guidelines, was considered
as the cause of not approving Anchor.
WILL; I RESPECT YOU, THOUGH FOR THE MOST PART YOU ARE
OVER THE TOP. WHAT I WROTE, IMO, IS FACT AND THE NEW GUIDLINES
DICTATED THE OUTCOME. YOU OUGHT TO CHECK OUT YOUR FACTS.
THE SPA IS BINDING ON THE SPONSORING CO, BUT THE FDA DOES
HAVE LEGAL WIGGLE ROOM. DID THEY TOTALLY MISHANFDLE ADCOM,
ABSOLUTELY ! HAD THE NEW AHA/ACA GUIDELINES COME OUT 6 WEEKS
LATER, ANCHOR WOULD HAVE BEEN APPROVED.
ZIP: You are absolutely correct. They completely mishandled ADCOM.
I have no idea why. I do know that the precipitating factor that
was a game changer. The FDA's being given the new ACA/AHA guidelines on approval of non statin lipid lowering agents. The
guidelines were specific in only using LDL C as a lipid Bio Marker
for approval, with an Outcomes Trial necessary but following approval. Triglycerides are not included ( NOT EVEN MENTIONED ONCE )in the new guidelines as an approvable lipid
metric for approval. Thus the Science changed and from what I know this has been the bottom line all the way up the FDA food change, on AMRN appeals. That's why AMRN has basically given up
on the Anchor Spa. A real shame and AMRN was totally blindsided.
FDA's reasoning is that they didn't have the new guidelines with
enough time to alert AMRN on the 75 day letter. For better or worse, AMRN got killed at the worst managed ADCOM I have been
witness to in 23 years.
JL: Sofinnova is a Ca based VC firm who follows VC
protocol. They were big sellers with JZ and pals
on 7-26-12 following the Marine approval. Most VC
models are no longer working. and their view on
publically traded stocks is to sell the news as
part of their exit strategy. Most of their capital,
about $1B, involves private Biotech Co's.
sts66: Hi, and how goes it? Greece and Spain are both
basically Socialistic nations whose politics have bankrupt
both Countries. I wouldn't exactly make them poster kids
for Eurozone drug pricing strategies. The Northern portion
of the Eurozone has been somewhat decent with their pricing.
Spain. Italy, and France are the top three in the race to
becoming Islamist States. France certainly deserves top billing.
biowreck: Because they are committed to. Less than that
would be prematurely throwing in the towel Don't forget
AMRN's NME gives them exclusivity until; 2017
HD: My point is that generic Spec Pharm Co's make a living
doing generics. Theoretically AZN could file , but that's not their Core focus. They are going to be very busy near term,
as I still believe PFE will acquire them
HD: If you think about it, AZN is not Spec Pharm
or in the business of making Generics. I worry
about the TEVA'S MYL, WATSON's et al. By the way,
V was listed in the Orange Book , but only after
Lovaza lost it's Orange Book listing , and at least
a year and a half after the 7-26-12 Marine approval.
HD: I believe in sweet justice.
Raf: I believe the FDA's bad behavior could allow
the Judge to do so. I'm not saying it's going to happen,
though the Judge is fully aware of both the punitive
actions on the part of Agency, as well as the lack of
receiving an NCE ruining any chance AMRN had of a BO
Raf: Correct. That said, I an hopeful that if the Judge
rules in AMRN favor, I believe we would get a new clock.
That would be an ideal outcome, and IMO a just one as well.
Raf: Exclusivity means just that. Generic makers can't
even think about filing for a generic if AMRN had an NCE.
The NCE protects the exclusivity for 5 years. After that
Generic makers can file for Generics, by attempting to
pick off what they perceive to be the weakest patents.
AMRN needs NCE as I don't have the same good feeling
re: patent protection as HD. (Though I hope I'm wrong)
Bottom line AMRN would spend most of their time in court
at a huge financial expense. The answer Biowreck gave you
is in point of fact incorrect.
Raf: Good question ! Not if AMRN should receive an
NCE or if their patent estate is as strong as they
claim. Personally I would prefer an NCE, not retroactive
to 7-12, when the Marine Indication was approved. Sadly,
that is in the FDA rule book, but IMO, the Judge has the
authority to attempt to make up the harm FDA caused AMRN,
by giving the NCE a new current date. I would not be happy
warding off the generic assault by depending on their existing
Patents. To my knowledge without a Composition of Matter Patent,
they would be in big trouble.
JL: Interesting historical tid bits. ESPR has an excellent
management team and a history of huge success. I owned
the stock back in the 2003-05 time frame. They had an
equally hot drug at that time that was an HDL Mimetic.
In a large PH II B trial they demonstrated a significant
decrease in Arterial Plaque, measured by intra arterial
ultrasonic pulse measurement. They promptly sold the drug
to PFE for as I recall $1.3B. I guess this is their encore.
Post Script: PFE took the drug into PH III trial which
unfortunately failed. These guys are extremely savvy. I
would wager they'll follow the same script.
Will: The decision was based simply on the new guidelines.
Triglycerides were not included. That is why AMRN dropped it.
Will: The answer to your question has been posted by me
on numerous occasions. The FDA had a few weeks prior access
of the change in AHA/ACA Guidelines where LDL C was considered
the only Lipid metric that could be approved prior to the
completion of an Outcome Trial.(PCSK9) Triglycerides were not included. ie, R-IT. FDA had access to these new guidelines a few weeks prior to our ADCOM meeting. They mishandled the ADCOM in an
inexcusable fashion. They maintained through the appeals process
all the way up the CDER food chain that the change in guidelines
represented a "New" or Change in the Science. AMRN had no other
recourse but to drop the Anchor Spa and pursue the NCE. For
shareholders this has been brutal, but apparently their argument
based on new science has been repeatedly upheld. The ruling sucks,
but does have scientific merit.
zum: Just to refresh your memory re PCSK9 drugs. There
was an agreement made between sponsoring Co's and Agency
following the new ACA/AHA guidelines naming LDL C as the
only lipid metric that was recognized for approval . This
presupposes that their endpoints are statistically significant
and demonstrated safety. Unfortunately Triglycerides were not
included and are required to wait for final results of Outcome
Trials. (R-IT) The PCSK9 drugs have to do Outcome Trials, but
will be approved prior to their completion. I posted this following the rescission of the AMRN Anchor SPA. IMO, the FDA
had access to the new guidelines 2 weeks prior to the Anchor
ADCOM and I fully believe was behind the FDA's destructive
decision. It basically represented a new change in Science.
KIWI; The 20 mg dose is used in approximately 80% of
patients on drug. High dose is used in subset groups
deemed to be at higher risk
north400000: I'm a bit short on time, but I will
give you few take away numbers from the AMGN abstract.
Patients on Evolocumab had a 53% reduction in CVE
versus standard of care, a 51% lowering of LDL and
an incidence of Neuro-Cognative AE of .9% . You might
want to pull up the AMGN abstract. These results were
at one year of therapy with Evolocumab.
Will: That BO happened 10 days ago, and
I believe ABBV is a great buy in here.
Contrary to the Streets saying they overpaid,
I believe Street numbers for Imbruvica are
$5B at peak sales. I model it at closer to
$12B. ABBV also got taken down because of a
possible Biosimilar for Humira, their $14.3B
RA drug. AMGN just saw a Biosimilar approved
for Neupogen, a very simple Biologic. Humira
is one of the most complex Biologics.I doubt
there will be a Biosimilar approved, in the
short term, if ever.
couldbebetter: I go back along time with them, and
IMO they are very smart guys. What individuates them
is they play with undervalued small cap company's, do very
thorough diligence, and are more often right than wrong.
They are probably more involved in SMID Cap Biotech (Small-
Mid Cap) than most Hedge Funds. I wouldn't be surprised that much of the recent increase in AMRN PPS, and volume is attributable
to other HF's piggy backing on their track record.
Will: My whole point all along is there is no precedent
for Congress attacking a retiring Agency Chief. With all
the hoopla surrounding the hijacking of the ADCOM , the
unprecedented rescission of the SPA, and a deliberate
hostility to AMRN, this is something that Agency has been
doing for as many years as I can recall. The SPA and AMRN
never even made the Congressional Agenda for the 3 sessions.
As for the PCSK9 drugs, they will shortly be on the market,
and because of superior efficacy in lowering LDL C, and most likely positive Outcome data, the 30 year old Statins (with
all their muscular skeletal side effects) will be used less and less, as the PCSK9 labels keep adding new indications. I just feel badly for the yeoman effort you put in to no avail. You were swinging at Windmills, and I hope it was Psycho-therapeutic.
JL: Nice to read your punditry again. FYI, two industry publications that came out this weekend are talking about
PFE interested in AMRN. Back to the future. Haven't heard
that in eons.
Kiwi: Great post, one of your best. I say that respectfully.
I just wanted you to know that my thoughts are with you with
the upcoming Angiogram. From where I sit, having a Stent put
in with your family history, should be looked as acceptable
maintenance, every 5-10 years, or as needed.
Hey Dude: You've got it almost figured out ! BP wasn't involved
in a mass corrupt operation. The FDA overplayed it's hand as it had privy to the revised changes in Lipid Guidelines made by the AHA/ACA. LDL was the only Lipid Biomarker accepted for FDA approval. Triglycerides were not included. A Non LDL Lipid Lowering Agent (Vascepa) was was not even considered for an approval until Reducing Trigs was proven effective in lowering CVD in an Outcome trial. R-IT. Ironically LDL lowering agents(PCKS9's)would be approved based just on statistically significant LDL reduction and safety. They would have to do Outcome Trials as well, albeit with they're drug already being marketed. It was the new guidelines that mucked things up for Vascepa and the rest is already known
Hey Dude: You've got it almost figured out ! BP wasn't involved
in a mass corrupt operation. The FDA overplayed it's hand as it had privy to the revised changes made by the AHA/ACA whereby LDL was the only Biomarker accepted for FDA approval. Triglycerides were not included . A Non LDL Lipid Lowering Agent (Vascepa) was
was not even considered for an approval until Reducing Trigs was
proven effective in an
Continued: I have a strong conviction that the real difference
maker is the anti inflammatory effect of the purified EPA on the Endothelial Cells lining the Coronary Arteries. I honestly feel it is a Class Wide effect, and includes all at risk, for MI and Stroke . As long as you can lower the CRP and the other three known Biomarkers for inflammation, you will have more positive
patient outcomes.
Kiwi: I have a different point of view about Vascepa. I don't
feel we need to identify sub sets of the at risk patients . Iction that the a